Key Themes

1. Tackling Pediatric-Specific Needs in Drug Development

Traditional approaches often do not meet the unique needs of paediatric populations, leading to challenges with dosage and potential side effects when adult drugs are adapted for children. The conferences emphasise the importance of customising research methodologies, clinical trial designs, and formulations to improve safety and efficacy in pediatric treatments.

2. Regulatory Advancements and Challenges

Key discussions addressed regulatory frameworks, including the Paediatric Regulation in the EU and similar initiatives in the U.S. Although these regulations have facilitated paediatric drug development, challenges remain in aligning regulatory requirements with the realities of conducting pediatric trials. Streamlining these processes was a prominent theme, aiming for better regulatory support tailored to paediatric needs.

3. The Power of Collaboration

The conferences showcased how multi-stakeholder collaboration across sectors—industry, academia, government, and patient organisations—drives innovation in pediatric medicine. Success stories highlighted at the events reveal that shared knowledge and resources are instrumental in addressing the logistical and financial challenges that paediatric research often faces.

4. Innovative Clinical Trial Models

With children, clinical trials bring unique ethical and logistical hurdles. Adaptive trials, real-world data, and age-appropriate designs were presented as promising solutions. These approaches balance ethical concerns with scientific rigor, ensuring that paediatric trials yield meaningful data without compromising young patients’ safety.

5. Future Directions and Ongoing Challenges

Topics like adolescent inclusion in adult trials, the role of real-world evidence, and optimising the development process were central to discussions of what comes next. The EU’s ongoing revision of pharmaceutical regulations—including the Paediatric and Orphan Regulations—reflects an evolving commitment to improving access and innovation in paediatric care.

Abstract

For many years, the European Forum for Good Clinical Practice (EFGCP) Children Medicines Working Party has organised a Paediatric conference annually. In the past, this event was organised jointly with the European Medicines Agency who was used to host it, along with the Drug Information Association (DIA). This conference is the opportunity for all involved in paediatric drug development, i.e., regulators, HTA bodies, patients’ representatives, academia and industry, to share learnings and raise awareness about new regulatory requirements of interest to optimise paediatric drug development.

The theme of the 2021 conference was “Challenges and Solutions – the path forward” while in 2022 it focused on “Progress made and Continuing Challenges”. Because of the COVID-19 pandemic these two conferences were organised virtually. However, this has not impacted the attendance and value of the conference, since because of a broad and attractive agenda there was a wide stakeholder participation, which provided a compendious overview of the leading issues to improve children’s access to innovative medicines.

For orally administered drugs, palatability is key in ensuring patient acceptability and treatment compliance. Therefore, understanding children’s taste sensitivity and preferences can support formulators in making paediatric medicines more acceptable.

Presently, we explore if the application of computer-vision techniques to videos of children’s reaction to gustatory taste strips can provide an objective assessment of palatability.

Primary school children tasted four different flavoured strips: no taste, bitter, sweet and sour. Data was collected at home, under the supervision of a guardian, with responses recorded using the Aparito Atom5™ app and smartphone camera.

Participants scored each strip on a 5-point hedonic scale. Facial landmarks were identified in the videos, and quantitative measures such as changes around the eyes, nose and mouth were extracted to train models to classify strip taste and score.

We received 197 videos and 256 self-reported scores from 64 participants. The hedonic scale elicited expected results: children like sweetness, dislike bitterness and have varying opinions for sourness. The findings revealed the complexity and variability of facial reactions and highlighted specific measures, such as eyebrow and mouth corner elevations, as significant indicators of palatability. This study into children’s taste specificities can improve the measurement of paediatric medicine acceptability.

An objective measure of how children feel about the taste of medicines has great potential in helping find the most palatable formulation.

Moreover, collecting data in the home setting allows for natural behaviour, with minimal burden for participants.

In this paper, our authors assess the feasibility of a digitized COA designed to video record the Timed up and Go (vTUG) test at home using the Atom5™ eCOA platform.

Background

Parkinson’s Disease (PD) is a progressive neurological disorder. Current therapeutic trials investigate treatments that can potentially modify the disease course. Testing their efficiency requires frequent and precise clinical outcome assessments (COA) of symptoms that remain problematic under symptomatic treatments, such as gait and balance. Home-based examinations may enhance patient compliance and, in addition, produce more reliable results by assessing patients more regularly in their familiar surroundings.

Objective

The objective of this study was to assess the feasibility of a digitized COA designed to video record the Timed up and Go (vTUG) test at home via a study-specific smartphone app for patients with PD.

Method

In this study, 28 patients were recruited and asked to perform at home each week a set of three consecutive vTUG tests over a period of 12 weeks using an app. The videos were subjected to a manual review to ascertain the durations of the individual vTUG phases, as well as to identify any errors or deviations in the setup that might have influenced the result. To evaluate the usability and user-friendliness of the vTUG and app, the System Usability Scale (SUS) and User Experience Questionnaire (UEQ) were administered to patients at the study end.

Access the paper and read the results

Video-based Clinical Outcome Assessments (vCOAs) are poised to revolutionize the management of pediatric rare diseases by providing a more accurate, engaging, and patient-centered approach to monitoring and evaluating treatment outcomes. These methods will become more integrated and essential in clinical trials and everyday care.

In this paper, our authors explore the advantages of vCOAs, the requirements for their implementation and validation, and what the future may hold for further vCOA development.

Abstract

Access the paper

Summary

The traditional 6-minute walk test (6MWT) has been widely used in clinical trials, but the authors suggest that a shorter test may be more practical, especially as clinical assessments move from hospital to community settings.

Their previous research shows that patients enthusiastically use digital and community-based exercise capacity tests.

The letter outlines efforts to design a community-based digital test that could provide similar diagnostic insights but with less burden on patients.

The study collected data from patients with PH and ILD, and early results have shown the practicality and patient acceptance of the 1MWT.

This innovative approach could help improve patient-centered care and outcomes in cardiorespiratory disease management.

Access the paper

Patient-reported outcomes (PROs) are crucial in healthcare research, offering insights into interventions from the patient’s perspective. This study focuses on mitigating respondent burden in #PROs to enhance data quality and decision-making.

After a thorough literature review and a Delphi survey involving multi-stakeholders, the authors compiled 19 recommendations to address this issue including “where possible, consider the use of ePROs, which may help reduce respondent burden, but must be balanced with the needs and preferences of the target population” and “explore the functionality of ePROs with diverse representatives from the target population where possible”.

Abstract

Access the paper